JP2020510424A5 - - Google Patents
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- Publication number
- JP2020510424A5 JP2020510424A5 JP2019546817A JP2019546817A JP2020510424A5 JP 2020510424 A5 JP2020510424 A5 JP 2020510424A5 JP 2019546817 A JP2019546817 A JP 2019546817A JP 2019546817 A JP2019546817 A JP 2019546817A JP 2020510424 A5 JP2020510424 A5 JP 2020510424A5
- Authority
- JP
- Japan
- Prior art keywords
- capsid protein
- modified
- pharmaceutical composition
- amino acid
- recombinant virus
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
- 102000004040 Capsid Proteins Human genes 0.000 claims description 54
- 108090000565 Capsid Proteins Proteins 0.000 claims description 54
- 241000700605 Viruses Species 0.000 claims description 35
- 230000003612 virological Effects 0.000 claims description 33
- 201000010099 disease Diseases 0.000 claims description 30
- 239000008194 pharmaceutical composition Substances 0.000 claims description 30
- 230000001225 therapeutic Effects 0.000 claims description 20
- 125000000539 amino acid group Chemical group 0.000 claims description 18
- 238000003780 insertion Methods 0.000 claims description 16
- 229920000023 polynucleotide Polymers 0.000 claims description 16
- 239000002157 polynucleotide Substances 0.000 claims description 16
- 210000004027 cells Anatomy 0.000 claims description 9
- 230000000875 corresponding Effects 0.000 claims description 9
- 239000000546 pharmaceutic aid Substances 0.000 claims description 8
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 7
- 206010012689 Diabetic retinopathy Diseases 0.000 claims description 6
- 208000009745 Eye Disease Diseases 0.000 claims description 6
- 102100006624 F9 Human genes 0.000 claims description 6
- 230000002265 prevention Effects 0.000 claims description 5
- 102000004169 proteins and genes Human genes 0.000 claims description 5
- 108090000623 proteins and genes Proteins 0.000 claims description 5
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 4
- 229940009550 C1 esterase inhibitor Drugs 0.000 claims description 4
- 108010041308 Endothelial Growth Factors Proteins 0.000 claims description 4
- 108010076282 Factor IX Proteins 0.000 claims description 4
- 229960000301 Factor VIII Drugs 0.000 claims description 4
- 108010054218 Factor VIII Proteins 0.000 claims description 4
- 102000001690 Factor VIII Human genes 0.000 claims description 4
- 102100019126 HBB Human genes 0.000 claims description 4
- 102100004899 HBG2 Human genes 0.000 claims description 4
- 108091005902 Hemoglobin subunit beta Proteins 0.000 claims description 4
- 229920001850 Nucleic acid sequence Polymers 0.000 claims description 4
- 229940024142 alpha 1-Antitrypsin Drugs 0.000 claims description 4
- 102000015395 alpha 1-Antitrypsin Human genes 0.000 claims description 4
- 108010050122 alpha 1-Antitrypsin Proteins 0.000 claims description 4
- 230000002137 anti-vascular Effects 0.000 claims description 4
- 239000003795 chemical substances by application Substances 0.000 claims description 4
- 229960004222 factor IX Drugs 0.000 claims description 4
- 108010038853 gamma-Globins Proteins 0.000 claims description 4
- 201000009673 liver disease Diseases 0.000 claims description 4
- 150000007523 nucleic acids Chemical group 0.000 claims description 4
- 208000004644 Retinal Vein Occlusion Diseases 0.000 claims description 3
- 206010038932 Retinopathy Diseases 0.000 claims description 3
- 206010038923 Retinopathy Diseases 0.000 claims description 3
- 229930002945 all-trans-retinaldehyde Natural products 0.000 claims description 3
- 230000002207 retinal Effects 0.000 claims description 3
- 235000020945 retinal Nutrition 0.000 claims description 3
- 239000011604 retinal Substances 0.000 claims description 3
- 241000432074 Adeno-associated virus Species 0.000 claims description 2
- 101700074227 F9 Proteins 0.000 claims description 2
- 208000009292 Hemophilia A Diseases 0.000 claims description 2
- 208000009429 Hemophilia B Diseases 0.000 claims description 2
- 229920002971 Heparan sulfate Polymers 0.000 claims description 2
- 210000003494 Hepatocytes Anatomy 0.000 claims description 2
- 206010019860 Hereditary angioedema Diseases 0.000 claims description 2
- 241000124008 Mammalia Species 0.000 claims description 2
- 230000035492 administration Effects 0.000 claims description 2
- 208000005980 beta-Thalassemia Diseases 0.000 claims description 2
- 238000002347 injection Methods 0.000 claims description 2
- 239000007924 injection Substances 0.000 claims description 2
- 230000000302 ischemic Effects 0.000 claims description 2
- 200000000011 liver disorder Diseases 0.000 claims description 2
- 239000012528 membrane Substances 0.000 claims description 2
- 201000007914 proliferative diabetic retinopathy Diseases 0.000 claims description 2
- 150000003839 salts Chemical class 0.000 claims description 2
- 239000011780 sodium chloride Substances 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 6
- 206010012601 Diabetes mellitus Diseases 0.000 claims 3
- 210000000234 Capsid Anatomy 0.000 claims 1
- 206010061992 Haemophilia Diseases 0.000 claims 1
- 206010030113 Oedema Diseases 0.000 claims 1
- 208000008425 Protein Deficiency Diseases 0.000 claims 1
- 210000001210 Retinal Vessels Anatomy 0.000 claims 1
- 206010038886 Retinal oedema Diseases 0.000 claims 1
- 230000033115 angiogenesis Effects 0.000 claims 1
- 201000005667 central retinal vein occlusion Diseases 0.000 claims 1
- 230000000938 luteal Effects 0.000 claims 1
- 201000011195 retinal edema Diseases 0.000 claims 1
- 201000001365 retinal ischemia Diseases 0.000 claims 1
- 239000000126 substance Substances 0.000 claims 1
- 239000002753 trypsin inhibitor Substances 0.000 claims 1
- 206010064930 Age-related macular degeneration Diseases 0.000 description 4
- 208000002780 Macular Degeneration Diseases 0.000 description 4
- 210000001957 Retinal Vein Anatomy 0.000 description 2
- 206010012688 Diabetic retinal oedema Diseases 0.000 description 1
- 102100000368 F8 Human genes 0.000 description 1
- 101700070229 F8 Proteins 0.000 description 1
- 206010029113 Neovascularisation Diseases 0.000 description 1
- 208000007135 Retinal Neovascularization Diseases 0.000 description 1
- 210000002966 Serum Anatomy 0.000 description 1
- 208000006682 alpha 1-Antitrypsin Deficiency Diseases 0.000 description 1
- 201000011190 diabetic macular edema Diseases 0.000 description 1
- 201000003542 factor VIII deficiency Diseases 0.000 description 1
- 230000000414 obstructive Effects 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762464878P | 2017-02-28 | 2017-02-28 | |
| US62/464,878 | 2017-02-28 | ||
| PCT/US2018/020215 WO2018160686A1 (en) | 2017-02-28 | 2018-02-28 | Modified aav capsids and uses thereof |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020510424A JP2020510424A (ja) | 2020-04-09 |
| JP2020510424A5 true JP2020510424A5 (es) | 2021-04-08 |
Family
ID=63370228
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019546817A Pending JP2020510424A (ja) | 2017-02-28 | 2018-02-28 | 改変ａａｖカプシドおよびその使用 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20210130413A1 (es) |
| EP (1) | EP3589277A4 (es) |
| JP (1) | JP2020510424A (es) |
| CN (1) | CN110650733A (es) |
| AU (1) | AU2018227483A1 (es) |
| CA (1) | CA3054687A1 (es) |
| WO (1) | WO2018160686A1 (es) |
Families Citing this family (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG10201810150UA (en) | 2014-03-17 | 2018-12-28 | Adverum Biotechnologies Inc | Compositions and methods for enhanced gene expression in cone cells |
| CN107405507B (zh) | 2015-03-02 | 2022-05-03 | 阿德夫拉姆生物技术股份有限公司 | 用于将多核苷酸玻璃体内递送到视网膜视锥的组合物和方法 |
| GB2545763A (en) | 2015-12-23 | 2017-06-28 | Adverum Biotechnologies Inc | Mutant viral capsid libraries and related systems and methods |
| EP3528785A4 (en) | 2016-10-19 | 2020-12-02 | Adverum Biotechnologies, Inc. | MODIFIED AAV CASPIDS AND USES THEREOF |
| EP3596213A4 (en) | 2017-03-17 | 2021-02-17 | Adverum Biotechnologies, Inc. | COMPOSITIONS AND PROCEDURES FOR ENHANCED GENE EXPRESSION |
| AU2018350990A1 (en) * | 2017-10-18 | 2020-05-21 | Regenxbio Inc. | Treatment of ocular diseases and metastatic colon cancer with human post-translationally modified VEGF-Trap |
| US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
| WO2020068990A1 (en) * | 2018-09-26 | 2020-04-02 | California Institute Of Technology | Adeno-associated virus compositions for targeted gene therapy |
| US20200297869A1 (en) * | 2019-03-04 | 2020-09-24 | Adverum Biotechnologies, Inc. | Sequential intravitreal administration of aav gene therapy to contralateral eyes |
| TW202102526A (zh) * | 2019-04-04 | 2021-01-16 | 美商銳進科斯生物股份有限公司 | 重組腺相關病毒及其用途 |
| AU2020257182A1 (en) * | 2019-04-15 | 2021-12-09 | Spirovant Sciences, Inc. | Methods and compositions for transgene expression |
| TW202106699A (zh) * | 2019-04-26 | 2021-02-16 | 美商愛德維仁生物科技公司 | 用於玻璃體內遞送之變異體aav蛋白殼 |
| CN111876432B (zh) * | 2020-07-29 | 2022-10-28 | 舒泰神（北京）生物制药股份有限公司 | 一组肝靶向新型腺相关病毒的获得及其应用 |
| CN113121652B (zh) * | 2021-04-19 | 2022-10-11 | 上海信致医药科技有限公司 | 视网膜和肌肉高亲和性腺相关病毒衣壳蛋白及相关应用 |
| CN114686448A (zh) * | 2022-03-31 | 2022-07-01 | 上海勉亦生物科技有限公司 | 具有肝脏特异靶向性的纯化的腺相关病毒及其应用 |
| CN112961220B (zh) * | 2021-04-19 | 2023-11-17 | 信念医药科技(上海)有限公司 | 腺相关病毒衣壳蛋白及包含其的腺相关病毒载体 |
| KR20240014477A (ko) | 2021-05-28 | 2024-02-01 | 상하이 레제네리드 테라피즈 컴퍼니 리미티드 | 변이체 캡시드를 갖는 재조합 아데노-연관 바이러스 및 이의 응용 |
| CN113564187A (zh) * | 2021-07-30 | 2021-10-29 | 上海信致医药科技有限公司 | 基于aav的抗血管生成基因递送系统及其用途 |
| CN117143920A (zh) * | 2023-08-30 | 2023-12-01 | 呈诺再生医学科技(北京)有限公司 | 一种治疗新生血管性眼部疾病的基因药物 |
| CN117368466B (zh) * | 2023-12-08 | 2024-03-12 | 安徽惠邦生物工程有限公司 | 用生化分析仪测定尿结合珠蛋白的试剂盒及其制备方法 |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PT1127150E (pt) * | 1998-11-05 | 2007-08-22 | Univ Pennsylvania | ''sequências de ácido nucleico do vírus adeno associado do serotipo 1, vectores e células hospedeiras que as contêm'' |
| US8663624B2 (en) * | 2010-10-06 | 2014-03-04 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| RS62795B1 (sr) * | 2011-04-22 | 2022-02-28 | Univ California | Adeno-povezani virioni virusa sa varijantama kapsida i postupci za njihovu primenu |
| GB201403260D0 (en) * | 2014-02-25 | 2014-04-09 | Univ Manchester | Treatment of retinal degeneration using gene therapy |
| WO2015138616A1 (en) * | 2014-03-11 | 2015-09-17 | Wayne State University | A modified mglur6 promoter and methods of use |
-
2018
- 2018-02-28 US US16/488,689 patent/US20210130413A1/en not_active Abandoned
- 2018-02-28 CN CN201880025324.7A patent/CN110650733A/zh active Pending
- 2018-02-28 WO PCT/US2018/020215 patent/WO2018160686A1/en unknown
- 2018-02-28 EP EP18760397.2A patent/EP3589277A4/en not_active Withdrawn
- 2018-02-28 AU AU2018227483A patent/AU2018227483A1/en not_active Abandoned
- 2018-02-28 CA CA3054687A patent/CA3054687A1/en active Pending
- 2018-02-28 JP JP2019546817A patent/JP2020510424A/ja active Pending
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